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The accessory makes the drug: ASO-delivered siRNA enters the human CNS for ALS

Wenxuan Guo1, Justin K Ichida1

  • 1Department of Stem Cell Biology and Regenerative Medicine, Keck School of Medicine, University of Southern California, Los Angeles, CA 90033, USA; Eli and Edythe Broad CIRM Center for Regenerative Medicine and Stem Cell Research at USC, Los Angeles, CA 90033, USA; Zilkha Neurogenetic Institute, Keck School of Medicine of the University of Southern California, Los Angeles, CA 90033, USA.

Cell Stem Cell
|October 1, 2026
PubMed

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